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Wednesday, October 3, 2012


Oct. 2, 2012, 7:37 a.m. EDT


By Saabira Chaudhuri

Cell Therapeutics Inc. said its drug Opaxio has been granted orphan-drug designation by the U.S Food and Drug Administration for the treatment of a malignant brain cancer.

Orphan-drug designation is granted by the FDA to novel drugs that seek to treat a rare disease or condition. The designation provides substantial potential benefits to the drug developer, including seven years of market exclusivity for the product upon regulatory approval, fee waivers and tax incentives.

Also called paclitaxel poliglumex, the drug received orphan-drug designation to treat glioblastoma multiforme. The designation was granted based on preliminary activity seen from results of Opaxio when added to standard therapy in a midstage trial.

Cell Therapeutics also said a randomized trial is underway for patients with the brain cancer with unmethylated MGMT comparing standard therapy and radiation to Opaxio and radiation.

Opaxio works by selectively enhancing the anti-tumor potency of radiation therapy in preclinical animal models. According to Cell, it has demonstrated "excellent tumor sensitization during clinical trials of locally advanced lower esophageal cancer."

In addition to the study in patients with glioblastoma multiforme, the treatment also is being tested in patients with locally advanced head and neck cancer in combination with another cancer drug.

The drug company cites data from the National Cancer Institute, which says glioblastoma multiforme is the most common and deadliest type of primary brain tumor in adults. It is estimated that there will be 10,000 to 12,000 new cases diagnosed in the U.S. this year.


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